EP 3956452 A4 20230315 - GENE THERAPIES FOR USHER SYNDROME (USH2A)
Title (en)
GENE THERAPIES FOR USHER SYNDROME (USH2A)
Title (de)
GENTHERAPIEN FÜR DAS USHER-SYNDROM (USH2A)
Title (fr)
THÉRAPIES GÉNIQUES POUR LE SYNDROME D'USHER (USH2A)
Publication
Application
Priority
- US 201962836429 P 20190419
- US 2020028487 W 20200416
Abstract (en)
[origin: WO2020214796A1] Aspects of the disclosure relate to compositions and methods useful for delivering minigenes to a subject. Accordingly, the disclosure is based, in part, on isolated nucleic acids and gene therapy vectors, such as viral (e.g., rAAV) vectors, comprising one or more gene fragments encoding a therapeutic gene product, such as a protein or peptide (e.g., a minigene). In some embodiments, the disclosure relates to gene therapy vectors encoding a USH2A protein (e.g., the gene product of USH2A gene) or a portion thereof. In some embodiments, compositions described by the disclosure are useful for treating diseases associated with mutations in the USH2A gene, for example Usher Syndrome.
IPC 8 full level
C12N 15/52 (2006.01); A61K 38/17 (2006.01); A61K 48/00 (2006.01); C07K 14/47 (2006.01); C12N 15/11 (2006.01); C12Q 1/68 (2006.01)
CPC (source: EP US)
A01K 67/0276 (2013.01 - EP); A61K 48/005 (2013.01 - EP); A61K 48/0058 (2013.01 - US); A61K 48/0075 (2013.01 - US); A61P 27/02 (2017.12 - US); C07K 14/47 (2013.01 - EP US); C12N 15/86 (2013.01 - US); A01K 2217/075 (2013.01 - EP); A01K 2227/105 (2013.01 - EP); A01K 2227/40 (2013.01 - EP); A01K 2267/0306 (2013.01 - EP); A61K 38/00 (2013.01 - EP); C12N 2750/14143 (2013.01 - EP US)
Citation (search report)
- [X] WO 2016005514 A1 20160114 - STICHTING KATHOLIEKE UNIV [NL]
- [X] WO 2014140051 A1 20140918 - FOND TELETHON [IT]
- [X] WO 2018055134 A1 20180329 - PROQR THERAPEUTICS II BV [NL]
- See references of WO 2020214796A1
Designated contracting state (EPC)
AL AT BE BG CH CY CZ DE DK EE ES FI FR GB GR HR HU IE IS IT LI LT LU LV MC MK MT NL NO PL PT RO RS SE SI SK SM TR
DOCDB simple family (publication)
WO 2020214796 A1 20201022; CN 114402075 A 20220426; EP 3956452 A1 20220223; EP 3956452 A4 20230315; US 2022202959 A1 20220630
DOCDB simple family (application)
US 2020028487 W 20200416; CN 202080044241 A 20200416; EP 20791702 A 20200416; US 202017604545 A 20200416